A Phase II Exploratory Study of SR604 Injection Evaluating the Safety and Efficacy in the Treatment of Congenital Coagulation Factor VII Deficiency

Официальное название

An Open-Label, Multicenter, Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetic Characteristics of SR604 Injection in Patients With Congenital Coagulation Factor VII Deficiency

Обзор

This is an open-label, multicenter, exploratory Phase II clinical trial designed to evaluate the efficacy, safety, and pharmacokinetic characteristics of SR604 Injection in patients with congenital coagulation Factor VII deficiency.

Вмешательства

• Препарат SR604
  SR604 will be administered as SC injection.

Первичные конечные точки

• Treated overall Annualized Bleeding Rate (ABR) [Срок оценки: Through 28 weeks of treatment.]

Вторичные конечные точки (8)

• Treated spontaneous annualized bleeding rate [Срок оценки: over 28 weeks of treatment]
• Treated overall annualized joint bleeding rate [Срок оценки: over 28 weeks of treatment]
• Overall annualized bleeding rate including treated and untreated bleeding events [Срок оценки: over 28 weeks of treatment,]
• Annualized menorrhagia bleeding rate (menstruating females only) [Срок оценки: over 28 weeks of treatment]
• Change in PBAC score (menstruating females only); [Срок оценки: From baseline over 28 weeks of treatment]
• Change in EQ-5D-5L health index score [Срок оценки: From pre-treatment over 28 weeks of treatment]
• Change in EQ-VAS score [Срок оценки: From baseline over 28 weeks of treatment;]
• Subject overall satisfaction score with treatment efficacy [Срок оценки: over 28 weeks of treatment]

Критерии участия

Критерии включения

• Age ≥12 years and ≤65 years at the time of signing the informed consent form; both genders eligible;
• Clinically diagnosed with congenital coagulation Factor VII deficiency, with historical or screening FVII activity <10%, and ≥2 treated new-onset bleeding events within 3 months prior to enrollment;
• No active bleeding symptoms prior to first administration of SR604 Injection;
• The subject and/or legal representative and impartial witness have signed the informed consent form, indicating voluntary agreement to participate in this trial, to provide biological samples for testing as required by the protocol, and to comply with the planned study visits;
• Female subjects (post-menarche) must have a negative serum pregnancy test (HCG) during the screening period; subjects with childbearing potential (females post-menarche or males post-spermarche) must agree to use highly effective contraceptive measures throughout the study period.

Критерии исключения

• Known history of hypersensitivity to the study drug formulation or any of its components;
• Intolerance to subcutaneous injection or other local skin abnormalities or dermatoses that may affect drug administration or safety assessment;
• Meeting any one of the following criteria during screening:

• Hemoglobin <60 g/L;
• Platelet count <100×10⁹/L;
• Abnormal hepatic or renal function: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥2.5× upper limit of normal (ULN), or total bilirubin ≥1.5× ULN; or serum creatinine (Cr) ≥1.5× ULN;
• Positive for anti-human immunodeficiency virus (HIV) antibodies.
• Any bleeding disorder other than congenital Factor VII deficiency, or other conditions causing significantly abnormal coagulation parameters (e.g., hemophilia A or B, von Willebrand disease, platelet disorders, vitamin K deficiency, etc.);
• Protein C deficiency or Protein S deficiency;
• History of thrombosis, family history of thrombosis, or history of thrombophilia;
• Intracranial hemorrhage within 2 years prior to signing the informed consent form;
• Severe cardiac disease, such as unstable angina, congestive heart failure (New York Heart Association class ≥III), serious arrhythmia (QTc interval >450 ms, corrected by Fridericia formula), uncontrolled hypertension (systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg), etc.;
• Female patients with menstrual abnormalities caused by organic gynecological conditions (e.g., uterine fibroids, endometriosis, adenomyosis, etc.);
• Received Factor VII-containing products within 48 hours prior to first administration of SR604 Injection; received whole blood or plasma transfusion within 2 weeks prior to first administration of SR604 Injection;
• Used any anticoagulants, antifibrinolytics, or agents affecting platelet function (including chemical drugs, biological products, or traditional Chinese medicine), including aspirin, within 1 week prior to screening, or required use of such agents during the treatment period;
• Underwent major surgery (defined as Grade III or IV surgery) within 1 month prior to signing the informed consent form, or planned to undergo surgery during the study period;
• Enrolled in other clinical trials within 1 month prior to signing the informed consent form;
• Miscarriage or pregnancy termination within 3 months prior to signing the informed consent form; pregnant or breastfeeding women;
• Mental illness or significant psychiatric disorder, or incapacity or lack of cognitive ability due to other causes;
• Other conditions deemed by the investigator to be unsuitable for enrollment, such as alcoholism, anticipated poor subject compliance preventing completion of dosing and study follow-up, poorly controlled comorbid chronic diseases, or serious systemic diseases.

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Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences — Тяньцзинь

Идентификаторы

NCT: NCT07711158 · LS-SR604-VII-II01

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗